CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF

    公开(公告)号:WO2020123410A1

    公开(公告)日:2020-06-18

    申请号:PCT/US2019/065294

    申请日:2019-12-09

    Applicant: AMGEN INC.

    Abstract: The present invention relates to chemically-modified RNAi constructs for reducing expression of a target gene. In particular, the invention relates to specific patterns of modified nucleotides to be incorporated into RNAi constructs to improve in vivo stability and efficacy. Also described are pharmaceutical compositions comprising the chemically-modified RNAi constructs and methods of inhibiting target gene expression in vivo by administering the chemically-modified RNAi constructs, for example, to treat or ameliorate various disease conditions.

    POTENT AND SELECTIVE INHIBITORS OF NAV1.3 AND NAV1.7
    9.
    发明申请
    POTENT AND SELECTIVE INHIBITORS OF NAV1.3 AND NAV1.7 审中-公开
    NAV1.3和NAV1.7的POTENT和选择性抑制剂

    公开(公告)号:WO2012125973A2

    公开(公告)日:2012-09-20

    申请号:PCT/US2012/029537

    申请日:2012-03-16

    Abstract: Disclosed is a composition of matter comprising an isolated polypeptide, which is a peripherally-restricted Nav1.7 inhibitor. In some disclosed embodiments, the isolated polypeptide is an inhibitor of Nav1.7 and/or Nav1.3. Other embodiments are conjugated embodiments of the inventive composition of matter and pharmaceutical compositions containing the inventive composition of matter. Isolated nucleic acids encoding some embodiments of inventive polypeptides and expression vectors, and recombinant host cells containing them are disclosed. A method of treating or preventing pain is also disclosed.

    Abstract translation: 公开了包含分离的多肽的物质组合物,其是外周限制的Nav1.7抑制剂。 在一些公开的实施方案中,分离的多肽是Nav1.7和/或Nav1.3的抑制剂。 其它实施方案是本发明组合物和包含本发明组合物的药物组合物的共轭实施方案。 公开了编码本发明多肽和表达载体的一些实施方案的分离的核酸,以及含有它们的重组宿主细胞。 还公开了治疗或预防疼痛的方法。

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